Featured Research

from universities, journals, and other organizations

Drug shows dramatic reduction in seizures in patients with tuberous sclerosis complex

Date:
July 15, 2013
Source:
Cincinnati Children's Hospital Medical Center
Summary:
A drug originally developed to prevent the rejection of transplanted organs has now been shown to dramatically reduce seizures in patients with tuberous sclerosis complex (TSC) -- a genetic disease characterized by benign tumors on multiple organ systems. TSC is estimated to affect more than a million individuals throughout the world.

A drug originally developed to prevent the rejection of transplanted organs has now been shown to dramatically reduce seizures in patients with tuberous sclerosis complex (TSC) -- a genetic disease characterized by benign tumors on multiple organ systems. TSC is estimated to affect more than a million individuals throughout the world.

Related Articles


The study is the latest to demonstrate the effectiveness of everolimus for TSC patients. Previous studies conducted at Cincinnati Children's Hospital Medical Center showed that everolimus reduced tumors in the brain and the kidney.

The newest study, led by a physician-scientist at Cincinnati Children's in collaboration with a team at Texas Children's Hospital in Houston, has been accepted by the journal Annals of Neurology, and is available online.

"Everolimus treatment reduced seizure frequency and duration in the majority of TSC epilepsy patients whose seizures previously did not respond to treatment," says Darcy Krueger, MD, PhD, a pediatric neurologist at Cincinnati Children's and lead author of the study. "This improvement in seizure control was associated with a better quality of life, and side effects were limited. Work is already underway to confirm these results in a follow-up, phase III clinical study."

"This has been positively life-changing for the patients involved and is nothing short of transformative in the treatment of epilepsy associated with cellular growth disorders, such as TSC," says Angus Wilfong, MD, director of the comprehensive epilepsy program at Texas Children's Hospital and associate professor of pediatrics and neurology at Baylor College of Medicine.

The study included 20 patients who were treated with everolimus. Their median age was 8. Half of the patients were enrolled at Cincinnati Children's and half at Texas Children's Hospital in Houston.

The researchers found that everolimus reduced seizure frequency by at least 50 percent in 12 of the 20 participants. The drug also reduced seizures in 17 of the 20 TSC patients by a median rate of 73 percent. Four patients were free of seizures and seven had at least a 90 percent reduction in seizure frequency.

Overall quality of life, as reported by the participants' parents, also improved. Parents reported several positive changes, including attention, behavior, social interaction and physical restrictions.

Studies in the 1990s traced the cause of TSC to defects in two genes, TSC1 and TSC2. When these genes malfunction, the cell has higher activity of mTOR, a protein known to trigger uncontrolled tumor cell and blood vessel growth. Everolimus shrinks tumors by inhibiting mTORC1, and it appears to reduce seizures in TSC patients in the same way.

"It is unclear whether the benefit of everolimus in treating epilepsy might extend beyond that observed in TSC," says Dr. Krueger. "mTORC1 has been implicated in genetic and neurodevelopmental syndromes in which epilepsy is prominent and in more common types of epilepsy. Additional clinical trials might tell us whether everolimus would benefit patients with epilepsy not related to TSC."

Funding for the study was provided by Novartis Pharmaceuticals and the Clack Foundation.


Story Source:

The above story is based on materials provided by Cincinnati Children's Hospital Medical Center. Note: Materials may be edited for content and length.


Journal Reference:

  1. Darcy A. Krueger, Angus A. Wilfong, Katherine Holland-Bouley, Anne E. Anderson, Karen Agricola, Cindy Tudor, Maxwell Mays, Christina M. Lopez, Mi-Ok Kim, David Neal Franz. Everolimus treatment of refractory epilepsy in tuberous sclerosis complex. Annals of Neurology, 2013; DOI: 10.1002/ana.23960

Cite This Page:

Cincinnati Children's Hospital Medical Center. "Drug shows dramatic reduction in seizures in patients with tuberous sclerosis complex." ScienceDaily. ScienceDaily, 15 July 2013. <www.sciencedaily.com/releases/2013/07/130715105121.htm>.
Cincinnati Children's Hospital Medical Center. (2013, July 15). Drug shows dramatic reduction in seizures in patients with tuberous sclerosis complex. ScienceDaily. Retrieved November 23, 2014 from www.sciencedaily.com/releases/2013/07/130715105121.htm
Cincinnati Children's Hospital Medical Center. "Drug shows dramatic reduction in seizures in patients with tuberous sclerosis complex." ScienceDaily. www.sciencedaily.com/releases/2013/07/130715105121.htm (accessed November 23, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, November 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Ebola-Hit Sierra Leone's Late Cocoa Leaves Bitter Taste

Ebola-Hit Sierra Leone's Late Cocoa Leaves Bitter Taste

AFP (Nov. 23, 2014) The arable district of Kenema in Sierra Leone -- at the centre of the Ebola outbreak in May -- has been under quarantine for three months as the cocoa harvest comes in. Duration: 01:32 Video provided by AFP
Powered by NewsLook.com
Don't Fall For Flu Shot Myths

Don't Fall For Flu Shot Myths

Newsy (Nov. 23, 2014) Misconceptions abound when it comes to your annual flu shot. Medical experts say most people older than 6 months should get the shot. Video provided by Newsy
Powered by NewsLook.com
WFP: Ebola Risks Heightened Among Women Throughout Africa

WFP: Ebola Risks Heightened Among Women Throughout Africa

AFP (Nov. 21, 2014) Having children has always been a frightening prospect in Sierra Leone, the world's most dangerous place to give birth, but Ebola has presented an alarming new threat for expectant mothers. Duration: 00:37 Video provided by AFP
Powered by NewsLook.com
Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins